BI’s Volasertib Is an Orphan Drug in the U.S. and EU

U.S. and EU regulators have both determined that Boehringer Ingelheim’s volasertib for acute myeloid leukemia is an orphan drug, the German drugmaker said Thursday. The designation, which is granted to drugs that serve patient populations of 200,000 or less in the U.S., or no more than five per every 10,000 in the EU, follows an FDA breakthrough therapy designation last year.

Because most AML patients are between the ages of 65 and 70 and have co-morbidities, many cannot tolerate the standard intensive chemotherapy and thus are left with poor outcomes. Volasertib, which works by blocking the cell cycle that in AML produces high rates of cell division, could provide “a new alternative for those patients who are currently left with limited options,” said CEO Klaus Dugi.

Volasertib is currently being evaluated with low-dose cytarabine chemotherapy in a Phase III study of treatment-naïve patients 65 years and older who are ineligible for remission induction therapy at 143 centers in North America, Latin America, Europe, South Africa and Asia.

The drugmaker’s oncology research focuses on angiogenesis inhibition, signal transduction inhibition and cell-cycle kinase inhibition. Volasertib is its most advanced pipeline compound for hematological cancers; that pipeline also includes BI 836858 for AML and BI 836826 for chronic lymphocytic leukemia and B-cell non-Hodgkin’s lymphoma. — Lena Freund

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